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基因治療學和RNAi 以基因為基之藥物開發

市場調查報告書

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1529409

CRISPR Cas9全球市場:市場機會及臨床試驗預測(2029年)

Global CRISPR Cas9 Market Opportunity & Clinical Trials Outlook 2029

出版日期: 2024年08月01日 | 出版商:

KuicK Research | 英文 180 Pages | 商品交期: 最快1-2個工作天內

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簡介目錄圖表

以前，包括化療和標靶治療在內的傳統治療方法被用來治療癌症、自體免疫疾病、發炎性疾病和其他疾病，但臨床研究人員一直在堅持不懈地致力於發現尖端技術的假設。 CRISPR/Cas9 療法的發現是由於遺傳疾病在世界範圍內的驚人傳播以及對有效治療的需求不斷增長。自 2024 年 7 月起，CRISPR/Cas9 療法已被批准用於治療 β 地中海貧血和鐮狀細胞性貧血，並在美國、歐盟、英國、沙烏地阿拉伯和巴林上市。

未來五年，第一波 CRISPR/Cas9 療法預計將獲得監管部門批准並進入市場。這是因為，隨著這些療法在現實環境中表現出有效性和安全性，這將為基於 CRISPR/Cas9 的療法的廣泛接受和應用鋪平道路。除此之外，這項突破性基因編輯技術的潛在應用將大大擴展到遺傳性疾病和癌症之外，擴展到心血管、神經退化性疾病和中樞神經系統疾病的治療。

迄今為止，CRISPR/Cas9療法的全球臨床管線逐年快速成長，多種療法進入臨床試驗，其他CRISPR/Cas9候選療法處於臨床前階段。許多正在進行的 CRISPR/Cas9 臨床試驗已達到開發後期階段，預計將在五到七年內滲透到市場。例如，Intellia Therapeutics 評估了單劑量NTLA-2001（一種CRISPR/Cas9 療法）與安慰劑相比，對轉甲狀腺素蛋白澱粉樣變性心肌病變(ATTR-CM) 患者的療效和安全性，我們已經啟動了一項多國III 期臨床試驗。該試驗（MAGNITUDE）將於 2023 年 12 月開始，預計 2028 年 4 月完成。

本報告提供全球CRISPR Cas9市場相關調查，提供市場概要，以及藥物趨勢，臨床試驗趨勢，提供著各地區趨勢，及加入此市場的主要企業競爭情形等資訊。

Global & Regional: Commercial & Clinical Outlook
Global CRISPR-Cas9 Therapy Clinical Trials By Company, County, Indication & Phase
Number Of CRISPR-Cas9 Therapies In Trials: > 25 Therapies
Approved CRISPR-Cas9 Therapy: Casgevy (Exagamglogene Autotemcel) Therapy Clinical Insight
Approved CRISPR-Cas9 Therapy Pricing & Dosing Analysis
Technology Platforms For Advancing CRISPR-Cas9 Therapy
Delivery Methodologies For CRISPR-Cas9
Competitive Landscape

Antecedently, out-of-date bestseller conventional therapies, including chemotherapy or targeted therapies, were utilized for the management of cancer, autoimmune, inflammation and other diseases; yet, clinical researchers were occupied tenaciously with the hypothesis of discovering other cutting edge technologies. The growing rampant of genetic disorders across the globe at a frightening gait as well as the need for an effective cure has led to the discovery of CRISPR/Cas9 therapy. Build on the triumph of gene therapies, these therapies clenches gargantuan plausibility with only one CRISPR/Cas9 therapy; Casgevy, approved into the commercial market, as of July 2024, to treat sickle cell anemia along with beta thalassemia and is currently accessible in US, EU, UK, Saudi Arabia and Bahrain; proposing novel opportunity in the approaching years.

With respect to KuicK Research statistics, we anticipate to perceive the first wave of CRISPR/Cas9 therapies receiving regulatory approvals over and above entering the market in upcoming 5 years due to the fact that as these therapies demonstrate their effectiveness in addition to safety in real-world settings, they will pave the way for broader acceptance and application of CRISPR/Cas9 based treatments. Coupled with this, the potential applications of this groundbreaking gene-editing technology are expected to expand dramatically beyond genetic disorders and cancers and will be utilized to cure cardiovascular, neurodegenerative together with CNS diseases as evident from rising preclinical as well as clinical studies, touching fundamentally every area of medicine and biotechnology.

Until now, the clinical pipeline for global CRISPR/Cas9 therapy is up surging year by year with multiple therapies have entered into clinical trials and other CRISPR/Cas9 candidates are lately in the preclinical stage. Many of ongoing CRISPR/Cas9 clinical trials have reached late stage of development and it is predicable that they will penetrate into the market in the imminent 5-7 years. For instance, Intellia Therapeutics has begun a phase III, multinational, multicenter, double-blind, placebo-controlled study is ongoing which aims to evaluate the efficacy and safety of a single dose of NTLA-2001, a CRISPR/Cas9 therapy, compared to placebo in participants with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The study (MAGNITUDE) was begun in December 2023 and is anticipated to be complete by April 2028 coupled with clenches an enrollment of 765 participants.

Just as importantly, the over-all market for CRISPR/Cas9 therapies is estimated to grow substantively and will become a multi-billion-dollar industry over the impending eons, driven by the snowballing number of preclinical and clinical trials, the likely regulatory approvals, in conjugation with the increase of indications. Currently, the US dominates the CRISPR/Cas9 sphere in terms of research and development activities, but EU is correspondingly far-sighted momentous evolution in this field.

As well as, the current approved CRISPR/Cas9 therapy; Casgevy, developed by CRISPR Therapeutics and Vertex Pharmaceutical, is launched into the market at an average cost of roughly US$ 2 Million per single course of treatment according to Casgevy developer company SEC filings. Additionally, the anticipated high prices of these therapies, justified by their potential curative nature, are expected to further drive significant market growth in terms of revenue, even with relatively small patient populations.

As a final point, all elucidations aforesaid epitomize that the global CRISPR/Cas9 therapy is growing at a trailblazing gait and is predictable to multiply additional in the forthcoming 10 years. The expanding clinical application as well as together with indication of CRISPR/Cas9, mounting preclinical and clinical trials, rise in technological advancement, presences of key players like Intellia Therapeutics, CRISPR Therapeutics, Editas Medicine, TransCode Therapeutics and many more, combination studies of CRISPR/Cas9 with other therapies coupled with augment in investment, collaborations and government bestow are driving the market penetration, offering opportunism to pharma and biotech companies in future.

1. Synopsis To CRISPR-Cas9 Therapy

1.1 Overview
1.2 Approved CRISPR-Cas9 Therapies

2. Delivery Methods For CRISPR-Cas9

2.1 Delivery of CRISPR-Cas9 Components Through Physical Methods
- 2.1.1 Microinjection
- 2.1.2 Electroporation
- 2.1.3 Hydrodynamic Delivery
2.2 Delivery of CRISPR-Cas9 Components through Viral Vectors
- 2.2.1 Adeno-Associated Virus (AAV)
- 2.2.2 Lentivirus & Adenovirus
2.3 Delivery of CRISPR-Cas9 Components through Non-Viral Vectors (Biomaterials)
- 2.3.1 Liposomes
- 2.3.2 Gold Nanoparticles
- 2.3.3 DNA Nanoclew

3. CRISPR-Cas9: A Home To Variety Of Applications

3.1 Advantages Accompanying CRISPR-Cas9 Technology
3.2 CRISPR-Cas9 Comparison with other Genome Editing Tools

4. Global CRISPR-Cas9 Therapy Clinical Trials Overview

4.1 By Company
4.2 By Country
4.3 By Indication
4.4 By Phase
4.5 By Priority Status

5. Global CRISPR-Cas9 Therapy Clinical Trials By Company, County, Indication & Phase

5.1 Research
5.2 Preclinical
5.3 Phase I
5.4 Phase I/II
5.5 Phase II

6. Approved CRISPR - Cas9 Therapy: Casgevy (Exagamglogene Autotemcel) Therapy Clinical Insight

7. Approved CRISPR-Cas9 Therapy - Overview, Pricing & Dosing Analysis

7.1 Casgevy (Exagamglogene Autotemcel)
- 7.1.1 Overview
- 7.1.2 Pricing & Dosing

8. Global CRISPR-Cas9 Therapy Current Market Trend & Developments

8.1 Current Market Outline
8.2 Future Market Outlook

9. CRISPR-Cas9 Therapy Market Trend Analysis By Region

9.1 US
9.2 UK
9.3 EU
9.4 China
9.5 Japan
9.6 South Korea

10. CRISPR-Cas9 Therapy Market Opportunities & Clinical Trial Statistics: By Indications

10.1 Inherited Blood Disorders
10.2 Genetic Disorders
10.3 Cancer
10.4 Autoimmune & Inflammatory Disorders
10.5 Microbial Infections
10.6 Cardiovascular Illnesses
10.7 Eye Disorders, Brain Diseases, Metabolic Illness & Other Conditions

11. Combination Approaches For CRISPR-Cas9 Therapy

12. Platforms For Advancing CRISPR-Cas9 Therapy

12.1 CRISPR-GNDM Modalis Therapeutics Platform
12.2 Intellia Therapeutics - Modular Platform
12.3 Editas Medicine CRISPR Gene Editing Platform
12.4 CRISPR-Cas9 Genome Editing Technology - ERS Genomics
12.5 CRISTARS Gene Editing Technology Innovation Platform- BRL Medicine
12.6 Gene Editing-BDmRNA Platform Shanghai BDgene
12.7 Kamau Therapeutics Next-Generation Gene Editing Platform
12.8 Excision BioTherapeutics CRISPR Gene Editing Technology

13. Global CRISPR-Cas9 Therapy Market Dynamics

13.1 Market Drivers
13.2 Market Achilles Heels

14. Competitive Landscape

14.1 4D Molecular Therapeutics
14.2 Arbor Biotechnologies
14.3 ASC Therapeutics
14.4 C4U Corporation
14.5 Casebia Therapeutics
14.6 CRISPR Therapeutics
14.7 Editas Medicine
14.8 Intellia Therapeutics
14.9 Nkarta Therapeutics
14.10 Vertex Pharmaceuticals

簡介目錄圖表

List of Tables

Table 1-1: Global - Approved CRISPR-Cas9 Therapies
Table 9-1: US - Ongoing CRISPR-Cas9 Clinical Trials
Table 9-2: CRISPR-Cas9 Candidates Granted FDA Designations
Table 9-3: EU - Ongoing Clinical Trials for CRISPR/Cas9-based Therapeutics, July'2024
Table 9-4: ERS Genomics - Collaborations
Table 10-1: Ongoing CRISPR-Cas9 Clinical Trials for Inherited Blood Disorders
Table 10-2: Ongoing CRISPR-Cas9 Clinical Trials for Cancer Disorders
Table 10-3: Collaboration Deals For Cancer CRISPR-Cas9 Therapy, July 2024
Table 11-1: Ongoing Preclinical & Clinical trials for Combination of CRISPR-Cas9 Therapy

List of Figures

Figure 1-1: CRISPR-Cas9 Therapy v/s Conventional Therapies
Figure 2-1: Delivery Methods for CRISPR-Cas9
Figure 2-2: Delivery Pathways for CRISPR-Cas9 Components
Figure 2-3: Physical Mode of Delivery for CRISPR-Cas9 Components
Figure 2-4: Viral Mode of Delivery for CRISPR-Cas9 Components
Figure 2-5: Non-Viral Mode of Delivery for CRISPR-Cas9 Components
Figure 3-1: Advantages of CRISPR-Cas9 Technology
Figure 3-2: Comparison between Different Genome Editing Tools
Figure 4-1: Global - CRISPR-Cas9 Therapy Clinical Trials by Company (Number of Drugs), 2024 till 2029
Figure 4-2: Global - CRISPR-Cas9 Therapy Clinical Trials by Country (Number of Drugs), 2024 till 2029
Figure 4-3: Global - CRISPR-Cas9 Therapy Clinical Trials by Indication (Number of Drugs), 2024 till 2029
Figure 4-4: Global - CRISPR-Cas9 Therapy Clinical Trials by Phase (Number of Drugs), 2024 till 2029
Figure 4-5: Global - CRISPR-Cas9 Therapy Clinical Trials by Priority Status (Number of Drugs), 2024 till 2029
Figure 7-1: Casgevy - Approval Year by Region
Figure 7-2: Casgevy - Patent Expiration Year, July'2024
Figure 7-3: US - Cost per Treatment of Casgevy (US$ in Million), July'2024
Figure 8-1: Global CRISPR-Cas9 Therapy - Important Companies
Figure 8-2: Dominating & Emerging Region in Global CRISPR-Cas9
Figure 8-3: CRISPR-Cas9 - Future Market
Figure 8-4: Global - CRISPR Cas9 Market Opportunity (US$ Million), 2024 -2029
Figure 9-1: CRISPR_SCD001 Phase I/II (NCT04774536) Study - Initiation & Completion Year
Figure 9-2: Intellia Therapeutics - In vivo & Ex vivo CRISPR Approach
Figure 9-3: Crucial US CRISPR-Cas9 Competitor
Figure 9-4: EBT-101 Phase I (NCT05144386) Study - Initiation & Completion Year
Figure 9-5: NTLA-2002 Phase I/II (NCT05120830) Study - Initiation & Completion Year
Figure 9-6: EU Major Hubs in CRISPR-Cas9 Therapy
Figure 9-7: BRL-101 Phase I (NCT06300723) Study - Initiation & Completion Year
Figure 9-8: ET-01 Phase I (NCT04925206) Study - Initiation & Completion Year
Figure 9-9: GRIT Biotechnology Clinical Pipeline
Figure 9-10: GT307 (NCT06453057) Study - Initiation & Completion Year
Figure 9-11: CRISPR-Cas9 - Key Players in China
Figure 9-12: Aspects Influencing Japanese CRISPR-Cas9 Market
Figure 9-13: Modalis Therapeutics & JCR Pharmaceuticals Collaboration
Figure 9-14: ToolGen's TGT-001 - Orphan Drug Designation
Figure 10-1: BRL-101 Phase I (NCT06287086) Study - Initiation & Completion Year
Figure 10-2: Acquisition of Graphite Bio Lead Candidate & CRISPR-Cas9 Platform by Kamau Therapeutics
Figure 10-3: Nula-cel Phase I/II (NCT04819841) Study - Initiation & Completion Year
Figure 10-4 REGV131-LNP1265 Phase I/II (NCT06379789) Study - Initiation & Completion Year
Figure 10-5: Sanofi & Scribe Therapeutics Collaboration
Figure 10-6: Discovery of Innovative CRISPR-Cas9 Approach by University of Trento
Figure 10-7: National Institutes of Health Grants for CRISPR-Cas9 Therapy
Figure 10-8: NTLA-2001 Phase I (NCT04601051) Study - Initiation & Completion Year
Figure 10-9: NTLA-2001 Phase III MAGNITUDE (NCT06128629) Study - Initiation & Completion Year
Figure 10-10: Commercial Genetic Disorder CRISPR-Cas9 Therapy Market Overview
Figure 10-11: CRISPR-Cas9 Approaches To Cure Cancer
Figure 10-12: CRISPR-Cas9 Combinations with Cryotherapy & Others For Cancer Treatment
Figure 10-13: CTX112 Phase I/II (NCT05643742) Study - Initiation & Completion Year
Figure 10-14: MEDiC Life Sciences Collaboration with Bristol Myers Squibb (BMS) on Tumor Target Discovery
Figure 10-15: Innovative Genomics Institute Gene Correction Strategy for Autoimmune Patients
Figure 10-16: BDgene Reported Optimistic Phase I Outcomes of BD111
Figure 10-17: CRISPR Therapeutics Pipeline
Figure 10-18: BD113vLVP (NCT06465537) Study - Initiation & Completion Year
Figure 10-19: ZVS203e Phase I (NCT05805007) Study - Initiation & Completion Year
Figure 10-20: Editas Medicine CRISPR-Cas9 Therapies Synopsis
Figure 10-21: EDIT-101 Phase I/II (NCT03872479) Study - Initiation & Completion Year
Figure 10-22: SNIPR Biome - CRISPR-Cas9 Engineered Microbial Gene Therapy For Various Indications
Figure 12-1: CRISPR-GNDM Modalis Therapeutics Platform
Figure 12-2: Advantage of CRISPR-GNDM
Figure 12-3: Editas Medicine - CRISPR Gene Editing Platform
Figure 12-4: Gene Editing Technology Innovation Platform (CRISTARS) - BRL Medicine
Figure 12-5: Gene Editing-BDmRNA Platform - BDgene
Figure 12-6: Kamau Therapeutics Next Generation Gene Editing Platform
Figure 12-7: Excision's CRISPR Approach
Figure 12-8: Excision BioTherapeutics CRISPR Gene Editing Technology
Figure 13-1: CRISPR-Cas9 Therapy - Market Drivers
Figure 13-2: CRISPR-Cas9 - Market Challenges